Clinical Trials

Question 1

Phase 1

Answer 1

Determines toxicity + establish maximum tolerated dose

Dose escalation is performed, starting at 10% of dose that is lethal in 10% of mice (LD10)

Question 2

Phase 1 pt requirements

Answer 2

Any tumour with no conventional therapy available

Pts must be fit + have normal hepatic + renal function

Question 3

Phase 2

Answer 3

Assesses particular anti-tumour activity in range of cancer
Measures response rate
Not necessary but useful to have a control arm + random allocation for treatment

Question 4

Phase 3

Answer 4

Randomised trials comparing new + established treatments

Assesses overall survival + progression free survival

Question 5

What are response end points?

Answer 5

Tumour growth/ shrinkage
Survival
Toxicity
Quality of life

Question 6

What is RECIST?

Answer 6

Response Evaluation Criteria in Solid Tumours

complete or partial response, progression or stable disease

Question 7

What statistical issues are involved with clinical trials?

Answer 7

Randomisation - reduces bias. Can be blinded or double blinded
Number of patients - to demonstrate a small effect at high significance, large cohort needed
Survival curves - data is presented graphically as a Kaplan Meier curve

Question 8

What is a basket trial?

Answer 8

Tumours harbouring genetic mutations are tested with new drug that targets mutation

Question 9

What is an umbrella trial?

Answer 9

Patients with particular type of cancer are tested to establish genetics of tumours + then offered different experimental treatments based on results

Question 10

What is a platform trial?

Answer 10

Multiple experimental arms are compared with single control arm to reduce costs